A Study to Evaluate Efficacy and Safety of AVTX-803 in Patients with Leukocyte Adhesion Deficiency Type II

Launched by AUG THERAPEUTICS · Jul 14, 2022

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called AVTX-803 for patients with a condition known as Leukocyte Adhesion Deficiency Type II (LAD II). This rare genetic disorder affects the immune system, making it harder for the body to fight infections. The main goal of the study is to see how effective and safe AVTX-803 is compared to stopping treatment altogether. The trial is currently recruiting participants aged between 6 months and 75 years who have been diagnosed with LAD II and have a history of infections that are difficult to treat.

If you or a family member are interested in participating, you would need to provide signed consent and, if applicable, assent if you are a minor. To be eligible, participants should not have severe anemia or kidney problems, and should not be pregnant. Those taking part can expect to follow specific study guidelines and use certain birth control methods if they are able to have children. This trial aims to provide valuable information that could lead to better treatment options for individuals with LAD II.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Subject must be between 6 months and 75 years old
• • Subject has biochemically and genetically proven LAD II (SLC35C1-CDG)
• • Subject has a documented history of Lewis antigen deficiency
• • Subject has a history of recurrent infections, opportunistic infections or infections that did not respond well to standard of care treatment
• • Subject or parent (for subjects under legal age for consent) has provided written informed consent for this study. Additionally, written informed assent has been provided, as appropriate, for minors of older age, per local institutional review board (IRB)/ethics committee (EC) policy and requirements
• • Subject is willing and able to comply with the protocol
• * Women of childbearing potential (WOCBP) meeting the criteria below:
• • 1. Non-lactating and has a negative pregnancy test at screening -AND-
• • 2. Uses an acceptable double-barrier method of contraception as determined by the investigator or sub-investigator for the duration of the study and 30 days following the last dose of study drug.
• • Male subjects must agree to use an acceptable double-barrier method of contraception with their partner as determined by the investigator or sub-investigator for the duration of the study and 30 days following the last dose of study drug.
• Exclusion Criteria:
• • Subject has severe anemia defined as hemoglobin \<8.0 g/dL (\<4.9 mmol/L)
• • Subject has impaired renal function as defined by an eGFR \<90 mL/min
• • Subject has a total absence of fucosylation on red blood cells and the presence of anti-H antigen
• • Subject has known or suspected intolerance or hypersensitivity to fucose or any ingredients of the investigational product
• • In the investigator's opinion, subject has a history of failure to respond to fucose at adequate dosing
• • In the investigator's opinion, subject is not able or not willing to comply with the study requirements.
• • Subject is pregnant